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WO2010085151A3 - Means and methods for modulating notch3 protein expression and/or the coding region of notch3; compositions and use thereof in the treatment of cadasil - Google Patents

Means and methods for modulating notch3 protein expression and/or the coding region of notch3; compositions and use thereof in the treatment of cadasil Download PDF

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Publication number
WO2010085151A3
WO2010085151A3 PCT/NL2010/050036 NL2010050036W WO2010085151A3 WO 2010085151 A3 WO2010085151 A3 WO 2010085151A3 NL 2010050036 W NL2010050036 W NL 2010050036W WO 2010085151 A3 WO2010085151 A3 WO 2010085151A3
Authority
WO
WIPO (PCT)
Prior art keywords
notch3
modulating
methods
coding region
cadasil
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Ceased
Application number
PCT/NL2010/050036
Other languages
French (fr)
Other versions
WO2010085151A2 (en
Inventor
Saskia Anne-Marie Jenny Lesnik Oberstein
Garrit-Jan Boudewijn Van Ommen
Adriana Marie Rus
Johannes Gregorius Dauwerse
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Leids Universitair Medisch Centrum LUMC
Original Assignee
Leids Universitair Medisch Centrum LUMC
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Leids Universitair Medisch Centrum LUMC filed Critical Leids Universitair Medisch Centrum LUMC
Priority to US13/145,873 priority Critical patent/US9062311B2/en
Priority to NZ594281A priority patent/NZ594281A/en
Priority to AU2010207073A priority patent/AU2010207073B8/en
Priority to CA2750584A priority patent/CA2750584A1/en
Priority to JP2011547840A priority patent/JP2012515547A/en
Priority to EP10702361.6A priority patent/EP2389390B1/en
Publication of WO2010085151A2 publication Critical patent/WO2010085151A2/en
Publication of WO2010085151A3 publication Critical patent/WO2010085151A3/en
Anticipated expiration legal-status Critical
Priority to US14/738,593 priority patent/US9353169B2/en
Ceased legal-status Critical Current

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Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/475—Growth factors; Growth regulators
    • C07K14/485—Epidermal growth factor [EGF], i.e. urogastrone
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70567—Nuclear receptors, e.g. retinoic acid receptor [RAR], RXR, nuclear orphan receptors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1138—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against receptors or cell surface proteins
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Toxicology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Microbiology (AREA)
  • Cell Biology (AREA)
  • Plant Pathology (AREA)
  • Immunology (AREA)
  • High Energy & Nuclear Physics (AREA)
  • Public Health (AREA)
  • Heart & Thoracic Surgery (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Animal Behavior & Ethology (AREA)
  • Vascular Medicine (AREA)
  • Cardiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Urology & Nephrology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)

Abstract

The invention among other provides means and methods for modulating NOTCH3 expression and/or protein coding domain. In one aspect the invention provides a method for at least reducing an elevated level of NOTCH3 protein in a NOTCH3 expressing cell or the immediate vicinity thereof said method comprising providing said cell with an anti-sense oligonucleotide specific for NOTCH3 m RNA or pre-m RNA thereby decreasing production of said NOTCH3 protein or thereby altering the protein coding region in said NOTCH3 m RNA or pre-m RNA.
PCT/NL2010/050036 2009-01-26 2010-01-26 Means and methods for modulating notch3 protein expression and/or the coding region of notch3; compositions and use thereof in the treatment of cadasil Ceased WO2010085151A2 (en)

Priority Applications (7)

Application Number Priority Date Filing Date Title
US13/145,873 US9062311B2 (en) 2009-01-26 2010-01-26 Means and methods for modulating NOTCH3 protein expression and/or the coding region of NOTCH3; compositions and use thereof in the treatment of CADASIL
NZ594281A NZ594281A (en) 2009-01-26 2010-01-26 Means and methods for modulating notch3 protein expression and/or the coding region of notch3; compositions and use thereof in the treatment of cadasil
AU2010207073A AU2010207073B8 (en) 2009-01-26 2010-01-26 Means and methods for modulating NOTCH3 protein expression and/or the coding region of NOTCH3; compositions and use thereof in the treatment of CADASIL
CA2750584A CA2750584A1 (en) 2009-01-26 2010-01-26 Means and methods for modulating notch3 protein expression and/or the coding region of notch3; compositions and use thereof in the treatment of cadasil
JP2011547840A JP2012515547A (en) 2009-01-26 2010-01-26 Means and methods for modulating NOTCH3 protein expression and / or the coding region of NOTCH3; its composition and use in the treatment of CADASIL
EP10702361.6A EP2389390B1 (en) 2009-01-26 2010-01-26 Means and methods for modulating notch3 protein expression and/or the coding region of notch3; compositions and use thereof in the treatment of cadasil
US14/738,593 US9353169B2 (en) 2009-01-26 2015-06-12 Means and methods for modulating NOTCH3 protein expression and/or the coding region of NOTCH3; compositions and use thereof in the treatment of CADASIL

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP09151326 2009-01-26
EP09151326.7 2009-01-26

Related Child Applications (2)

Application Number Title Priority Date Filing Date
US13/145,873 A-371-Of-International US9062311B2 (en) 2009-01-26 2010-01-26 Means and methods for modulating NOTCH3 protein expression and/or the coding region of NOTCH3; compositions and use thereof in the treatment of CADASIL
US14/738,593 Division US9353169B2 (en) 2009-01-26 2015-06-12 Means and methods for modulating NOTCH3 protein expression and/or the coding region of NOTCH3; compositions and use thereof in the treatment of CADASIL

Publications (2)

Publication Number Publication Date
WO2010085151A2 WO2010085151A2 (en) 2010-07-29
WO2010085151A3 true WO2010085151A3 (en) 2010-11-25

Family

ID=40578168

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/NL2010/050036 Ceased WO2010085151A2 (en) 2009-01-26 2010-01-26 Means and methods for modulating notch3 protein expression and/or the coding region of notch3; compositions and use thereof in the treatment of cadasil

Country Status (7)

Country Link
US (2) US9062311B2 (en)
EP (1) EP2389390B1 (en)
JP (2) JP2012515547A (en)
AU (1) AU2010207073B8 (en)
CA (1) CA2750584A1 (en)
NZ (1) NZ594281A (en)
WO (1) WO2010085151A2 (en)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
HK1211322A1 (en) * 2013-03-15 2016-05-20 昂考梅德药品有限公司 Methods of treating pancreatic cancer
US10407500B2 (en) * 2014-09-25 2019-09-10 INSERM (Institut National de la Santé et de la Recherche Médicale) Immunological treatment of cerebral autosomal dominant arteriopathy with subcortical infarcts and leukoencephalopathy
EP3207136A1 (en) * 2014-10-17 2017-08-23 Nogra Pharma Limited Methods and compositions for treating a subject with a smad7 antisense oligonucleotide
CN106148504A (en) * 2015-04-23 2016-11-23 上海交通大学医学院附属第九人民医院 The detection primer of dominant heredity EEG source location related gene and kit and purposes
CN114929336A (en) * 2019-09-19 2022-08-19 阿尔奈科学有限责任公司 Compounds and methods for modulating gene splicing
EP4347634A1 (en) * 2021-06-02 2024-04-10 Regeneron Pharmaceuticals, Inc. Treatment of cerebrovascular disease with neurogenic locus notch homolog protein 3 (notch3) agents
CN114062682A (en) * 2021-11-10 2022-02-18 厦门大学附属心血管病医院 Use of NOTCH3 as a detection target
CN115820733B (en) * 2022-11-11 2024-12-17 北京希诺谷生物科技有限公司 Method for establishing CADAIL disease model dogs
CN119889439B (en) * 2024-12-09 2025-11-04 安徽大学 A method and system for predicting protein necessity in different cell lines

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20030186290A1 (en) * 1996-08-01 2003-10-02 Institut National De La Sante Et De La Recherche Medicale (Inserm) Gene involved in CADASIL, method of diagnosis and therapeutic application
US20040102390A1 (en) * 2002-11-21 2004-05-27 Isis Pharmaceuticals Inc. Modulation of Notch3 expression

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20030186290A1 (en) * 1996-08-01 2003-10-02 Institut National De La Sante Et De La Recherche Medicale (Inserm) Gene involved in CADASIL, method of diagnosis and therapeutic application
US20040102390A1 (en) * 2002-11-21 2004-05-27 Isis Pharmaceuticals Inc. Modulation of Notch3 expression

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
FEDERICO A ET AL: "The spectrum of mutations for CADASIL diagnosis", NEUROLOGICAL SCIENCES ; OFFICIAL JOURNAL OF THE ITALIAN NEUROLOGICAL SOCIETY, SPRINGER-VERLAG, MI, vol. 26, no. 2, 1 June 2005 (2005-06-01), pages 117 - 124, XP019361567, ISSN: 1590-3478 *
SAIKI S ET AL: "Varicose veins associated with CADASIL result from a novel mutation in the Notch3 gene.", NEUROLOGY 25 JUL 2006, vol. 67, no. 2, 25 July 2006 (2006-07-25), pages 337 - 339, XP002526569, ISSN: 1526-632X *
VAN OMMEN GERT-JAN ET AL: "The therapeutic potential of antisense-mediated exon skipping", CURRENT OPINION IN MOLECULAR THERAPEUTICS, CURRENT DRUGS, LONDON, GB, vol. 10, no. 2, 1 April 2008 (2008-04-01), pages 140 - 149, XP009107079, ISSN: 1464-8431 *

Also Published As

Publication number Publication date
US9062311B2 (en) 2015-06-23
JP2012515547A (en) 2012-07-12
NZ594281A (en) 2013-09-27
AU2010207073B8 (en) 2017-05-18
WO2010085151A2 (en) 2010-07-29
US20150274796A1 (en) 2015-10-01
AU2010207073A1 (en) 2011-08-18
EP2389390B1 (en) 2020-11-04
AU2010207073A8 (en) 2017-05-18
EP2389390A2 (en) 2011-11-30
US20110318787A1 (en) 2011-12-29
JP2017148053A (en) 2017-08-31
CA2750584A1 (en) 2010-07-29
AU2010207073B2 (en) 2017-05-11
US9353169B2 (en) 2016-05-31

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